Belzutifan 是一种缺氧诱导因子 2 α 抑制剂，最初被批准用于 von Hippel-Lindau 病患者，最近根据 LITESPARK-005 的结果，被批准用于散发性转移性透明细胞肾细胞癌 (ccRCC)。关于 Belzutifan 在散发性、转移性 ccRCC 患者中的实际使用经验的数据很少。本研究旨在描述散发性转移性 ccRCC 患者使用 Belzutifan 的临床结果。对 22 名在 FDA 批准之前在 MD 安德森癌症中心接受 Belzutifan 治疗的患者进行了回顾性研究。无进展生存期 (PFS) 和客观缓解率 (ORR) 由盲法放射科医生使用实体瘤缓解评估标准 (RECIST) 1.1 版进行评估。 PFS 和总生存期 (OS) 从开始使用 belzutifan 时开始测量。中位随访时间为 14.9 个月。大多数患者患有国际转移性肾细胞癌数据库联盟中度风险疾病，超过三个转移部位，并且在开始使用belzutifan时中位数为五线既往治疗；所有患者均接受过免疫检查点治疗（ICT）和血管内皮生长因子受体酪氨酸激酶抑制剂（VEGFR-TKI）。中位 PFS 为 8.51 个月（95% 置信区间 [CI] 0-18.4），ORR 为 36.4%。中位 OS 为 14.72 个月 (95% CI 7.34-22.10)。在 22 名患者中，4 名 (18.2%) 患者需要减少剂量，3 名 (13.6%) 患者因药物不良事件 (ADE) 停用贝祖替凡。最常见的 ADE 是贫血（77.3%；17/22）和缺氧（36.4%；8/22）。没有发生与治疗相关的死亡。在经过大量预先治疗的散发性转移性 ccRCC 患者队列中，belzutifan 具有有意义的临床活性并且耐受性良好。这些真实世界的结果补充了 LITESPARK-005 的结果，并支持在 ICT 和 VEGFR-TKI 进展后使用 belzutifan。 Belzutifan 是一种新药，用于治疗一种已扩散到其他部位的透明细胞肾癌身体（已转移）。 MD 安德森癌症中心的一项研究跟踪了 22 名接受 Belzutifan 治疗的患者，发现它可以在近 9 个月的时间内控制癌症，并使 36% 的患者的癌症缩小。这项研究证实，即使在其他治疗无效后，belzutifan 也可以有效且安全。版权所有 © 2024 欧洲泌尿外科协会。由 Elsevier B.V. 出版。保留所有权利。

Belzutifan, a hypoxia-inducible factor 2 alpha inhibitor, was approved initially for patients with von Hippel-Lindau disease and more recently for sporadic, metastatic clear cell renal cell carcinoma (ccRCC) based on the results of LITESPARK-005. There is a paucity of data regarding real-world experience with belzutifan in patients with sporadic, metastatic ccRCC. This study aims to describe clinical outcomes with belzutifan in patients with sporadic, metastatic ccRCC.A retrospective study of 22 patients who received belzutifan at MD Anderson Cancer Center prior to the Food and Drug Administration approval was conducted. Progression-free survival (PFS) and objective response rate (ORR) were assessed by a blinded radiologist using Response Evaluation Criteria In Solid Tumors (RECIST) version 1.1. PFS and overall survival (OS) were measured from belzutifan initiation.The median follow-up time was 14.9 mo. Most patients had International Metastatic RCC Database Consortium intermediate-risk disease, more than three metastatic sites, and a median of five prior lines of treatment at initiation of belzutifan; all patients received prior immune checkpoint therapy (ICT) and vascular endothelial growth factor receptor tyrosine kinase inhibitors (VEGFR-TKIs). The median PFS was 8.51 mo (95% confidence interval [CI] 0-18.4) and ORR was 36.4%. The median OS was 14.72 mo (95% CI 7.34-22.10). Of 22 patients, four (18.2%) patients required dose reductions and three (13.6%) patients discontinued belzutifan because of adverse drug events (ADEs). The most common ADEs were anemia (77.3%; 17/22) and hypoxia (36.4%; 8/22). There were no treatment-related deaths.In a heavily pretreated cohort of patients with sporadic, metastatic ccRCC, belzutifan had meaningful clinical activity and was well tolerated. These real-world results add to the results of LITESPARK-005 and support the use of belzutifan after progression on ICT and VEGFR-TKIs.Belzutifan is a new medicine used to treat a type of clear cell kidney cancer that has spread to other parts of the body (metastasized). A study at MD Anderson Cancer Center followed 22 patients who were treated with belzutifan, and found that it worked to control the cancer for almost 9 mo and caused the cancer to shrink in 36% of patients. This study confirms that belzutifan can be effective and safe, even after other treatments have not worked.Copyright © 2024 European Association of Urology. Published by Elsevier B.V. All rights reserved.